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What are the goals of the Phase 2a hemophilia trial of ataluren?

April 26, 2017ataluren goals hemophilia phase trial

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What are the goals of the Phase 2a hemophilia trial of ataluren?

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The main goals of this trial are to determine whether ataluren can result in an increase in Factor VIII or IX levels and whether the drug can safely be given to people with severe hemophilia due to a nonsense mutation.